MX2009011055A - Tratamiento de la enfermedad de gaucher con chaperonas farmacologicas especificas y supervision del tratamiento utilizando marcadores sustitutos. - Google Patents

Tratamiento de la enfermedad de gaucher con chaperonas farmacologicas especificas y supervision del tratamiento utilizando marcadores sustitutos.

Info

Publication number
MX2009011055A
MX2009011055A MX2009011055A MX2009011055A MX2009011055A MX 2009011055 A MX2009011055 A MX 2009011055A MX 2009011055 A MX2009011055 A MX 2009011055A MX 2009011055 A MX2009011055 A MX 2009011055A MX 2009011055 A MX2009011055 A MX 2009011055A
Authority
MX
Mexico
Prior art keywords
treatment
gaucher disease
surrogate markers
specific pharmacological
pharmacological chaperones
Prior art date
Application number
MX2009011055A
Other languages
English (en)
Spanish (es)
Inventor
David Lockhart
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of MX2009011055A publication Critical patent/MX2009011055A/es

Links

Classifications

    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/92—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving lipids, e.g. cholesterol, lipoproteins, or their receptors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2405/00—Assays, e.g. immunoassays or enzyme assays, involving lipids
    • G01N2405/08—Sphingolipids
    • G01N2405/10—Glycosphingolipids, e.g. cerebrosides, gangliosides
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Urology & Nephrology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Neurology (AREA)
  • Biophysics (AREA)
  • Analytical Chemistry (AREA)
  • Pathology (AREA)
  • Biotechnology (AREA)
  • Cell Biology (AREA)
  • General Physics & Mathematics (AREA)
  • Endocrinology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Food Science & Technology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pain & Pain Management (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MX2009011055A 2007-04-13 2008-04-11 Tratamiento de la enfermedad de gaucher con chaperonas farmacologicas especificas y supervision del tratamiento utilizando marcadores sustitutos. MX2009011055A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US91169907P 2007-04-13 2007-04-13
US2812308P 2008-02-12 2008-02-12
PCT/US2008/060116 WO2008128106A1 (en) 2007-04-13 2008-04-11 Treatment of gaucher disease with specific pharmacological chaperones and monitoring treatment using surrogate markers

Publications (1)

Publication Number Publication Date
MX2009011055A true MX2009011055A (es) 2010-01-13

Family

ID=39864349

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2009011055A MX2009011055A (es) 2007-04-13 2008-04-11 Tratamiento de la enfermedad de gaucher con chaperonas farmacologicas especificas y supervision del tratamiento utilizando marcadores sustitutos.

Country Status (7)

Country Link
US (1) US8399525B2 (ja)
EP (1) EP2139323B1 (ja)
JP (1) JP5586453B2 (ja)
AU (1) AU2008240138B2 (ja)
CA (1) CA2683713A1 (ja)
MX (1) MX2009011055A (ja)
WO (1) WO2008128106A1 (ja)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2659904T3 (pl) 2008-06-26 2016-01-29 Orphazyme Aps Zastosowanie Hsp70 jako regulatora aktywności enzymatycznej
PL2646044T3 (pl) 2010-11-30 2020-03-31 Orphazyme A/S Sposoby zwiększenia aktywności wewnątrzkomórkowej Hsp70
PL3366688T3 (pl) 2010-12-08 2022-05-23 The U.S.A. As Represented By The Secretary, Department Of Health And Human Services Podstawione pirazolopirymidyny jako aktywatory glukocerebrozydazy
EP2533051A1 (en) * 2011-06-06 2012-12-12 Centogene GmbH Method for the diagnosis of Gaucher's disease
EP3922242A1 (en) 2014-09-15 2021-12-15 Orphazyme A/S Arimoclomol formulation
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
EP3782624A1 (en) 2016-04-29 2021-02-24 Orphazyme A/S Arimoclomol for treating glucocerebrosidase associated disorders
WO2017205451A1 (en) 2016-05-24 2017-11-30 Temple University-Of The Commonwealth System Of Higher Education Novel functionalized n,n-dialkylamino phenyl ethers and their method of use
WO2019173283A1 (en) 2018-03-05 2019-09-12 Marquette University Method and apparatus for non-invasive hemoglobin level prediction
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
EP4247792A1 (en) 2020-11-19 2023-09-27 Zevra Denmark A/S Processes for preparing arimoclomol citrate and intermediates thereof
EP4251140A1 (en) * 2020-11-25 2023-10-04 Lysosomal and Rare Disorders Research and Treatment Center, Inc. Inhibition of caspase pathway as a treatment for lysosomal storage disorders

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
AU2004289327A1 (en) 2003-11-12 2005-05-26 Amicus Therapeutics Inc. Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat Gaucher disease
AU2006254796B2 (en) * 2005-06-08 2012-08-16 Amicus Therapeutics, Inc. Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes

Also Published As

Publication number Publication date
AU2008240138A1 (en) 2008-10-23
US8399525B2 (en) 2013-03-19
EP2139323A4 (en) 2010-04-21
AU2008240138B2 (en) 2013-03-07
JP2010523715A (ja) 2010-07-15
EP2139323B1 (en) 2013-08-21
WO2008128106A1 (en) 2008-10-23
US20100196279A1 (en) 2010-08-05
JP5586453B2 (ja) 2014-09-10
EP2139323A1 (en) 2010-01-06
CA2683713A1 (en) 2008-10-23

Similar Documents

Publication Publication Date Title
MX2009011055A (es) Tratamiento de la enfermedad de gaucher con chaperonas farmacologicas especificas y supervision del tratamiento utilizando marcadores sustitutos.
EP2255263A4 (en) REACHEMINER WITH TWO REFERENCE CLOCKS AND ITS ACTUATION METHOD
IL195069A0 (en) Method and apparatus for the management of battery cells
MX2012001310A (es) Metodos para proporcionar efectos beneficos en la cavidad bucal.
EG25676A (en) Wall block with weight bearing pads and method of producing wall blocks
IL204628A0 (en) Wall block with weight bearing pads and method of producing wall blocks
EP2619168A4 (en) METHOD AND APPARATUS FOR ELECTROCHEMICAL REDUCTION OF CARBON DIOXIDE
EP2106874A4 (en) METHOD FOR PROCESSING ROCK WITH A LASER AND DEVICE THEREFOR
SG10201405380QA (en) Endocrine precursor cells, pancreatic hormone-expressing cells and methods of production
EP2335060A4 (en) ELECTROCHEMICAL BIOSENSOR STRUCTURE AND METHOD OF MEASUREMENT THEREWITH
IL182940A0 (en) Solid electrolyte and method of producing the same
EP2283559A4 (en) SYSTEM AND METHODS FOR EXTENDING SERVICE LIFE OF PORTABLE DEVICES
PL2206215T3 (pl) Sposób i urządzenie do wyznaczania rezonansu akumulatora
EP2286479A4 (en) BATTERY PARTS AND SAME MANUFACTURING AND USE METHOD
EP2281881A4 (en) HEFMUTANTE AND SUBSTANCE MANUFACTURING METHOD USING THEREOF
WO2009114679A3 (en) Treatment of pompe disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
IL255914B (en) A method that includes testing cells for the expression and/or secretion of one or more pro-angiogenic factors
FI20075860A (fi) Menetelmä ja järjestely elektrolyysialtaan elektrolyysiprosessin tilan seuraamiseksi ja esittämiseksi
TWI349818B (en) Repairing method and structure of display electrode
GB0608092D0 (en) Rock bolt and method of use
FI20075962A (fi) Menetelmä ja laitteisto pienpanoslouhintaan
AU312804S (en) Detachable cable manager
FI20080249A0 (fi) Mikrobiologinen tuotantomenetelmä ja laitteisto sen käyttämiseksi
EP2063850A4 (en) VIPR1 AS MODIFIERS OF THE E2F / RB PATHWAY AND METHODS OF USE
UA37789U (uk) Спосіб кріплення закладної деталі в отвір у бетонній плиті

Legal Events

Date Code Title Description
FG Grant or registration